burosumab (Crysvita)

following reassessment through the ultra-orphan framework:

burosumab (Crysvita®) is accepted for use within NHSScotland.

Indication under review: for the treatment of X-linked hypophosphataemia in children and adolescents aged 1 to 17 years with radiographic evidence of bone disease.
In an open-label, randomised, phase III study in patients aged 1 to 12 years with X-linked hypophosphataemia, there was a significantly greater improvement in rickets, assessed by the Radiographic Global Impression of Change global score at week 40, in the burosumab group compared with the conventional therapy group (oral phosphate and vitamin D).

In addition, the company provided further data from extension phases of the main studies and some supportive observational data on the use of burosumab in patients with X-linked hypophosphataemia.

This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/ list price that is equivalent or lower.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.